• Home
• Our Motivation
  • Patient Stories
    • Annabelle
    • Isabel
    • Matty
    • Miller
    • Sarah
  • Diseases & Conditions
    • Hemophilia A
    • Achondroplasia
    • PKU
    • CLN2 Disease
    • MPS VI
    • MPS IVA
    • MPS I
  • Patient & Community Support
    • Trees of Life
    • RARE Scholars
    • In the Hemophilia Community
    • Patient Advocacy
    • 澳门正规博彩十大排行平台 RareConnections™
    • Grants & Donations
  • Resource Library
    • Patient Organizations
    • Professional Organizations
• Our Science
  • Our Approach
    • Technical Expertise
    • Indication Expansion
    • Franchise Building
  • Our Scientists
  • Our Publications
  • Science Spotlight
    • 澳门正规博彩十大排行平台 Scientist Spotlight: Drs. Sylvia Fong & Soumi Gupta Discuss the Significance of AAV Therapies
    • Advancing our understanding of gene therapy research, one publication at a time
    • Meet the Scientist: Christian Vettermann, Ph.D.
    • 澳门正规博彩十大排行平台’s Center of Technology empowers scientists with technology, resources, and collaboration
  • Funding & Support
    • Independent Medical Education
    • Independent & Collaborative Research
    • Publication Data Request
• Our Treatments
  • Our Products
    • ROCTAVIAN™ (valoctocogene roxaparvovec-rvox)
    • VOXZOGO^® (vosoritide)
    • PALYNZIQ^® (pegvaliase-pqpz) Injection
    • BRINEURA^® (cerliponase alfa)
    • VIMIZIM^® (elosulfase alfa)
    • KUVAN^® (sapropterin dihydrochloride)
    • NAGLAZYME^® (galsulfase)
    • ALDURAZYME^® (laronidase)
  • Our Pipeline
  • Compassionate Use Policy
• Our Company
  • About Us
    • Our History
    • Manufacturing Excellence
    • Message from Chairman and CEO
    • Global Compliance & Ethics
  • Corporate Social Responsibility
    • Diversity, Equity & Inclusion
    • Environmental Health, Safety & Sustainability (EHS&S)
    • Environment, Social & Governance (ESG)
  • Leadership Team
    • Jean-Jacques Bienaimé
    • Henry J. Fuchs, M.D.
    • Jeff Ajer
    • Brinda Balakrishnan, M.D., Ph.D.
    • G. Eric Davis
    • C. Greg Guyer, Ph.D.
    • Marni Kottle
    • Philip Lo Scalzo
    • Brian R. Mueller
    • Amy Wireman
  • Board of Directors
  • Funding & Support
    • Independent Medical Education
    • Independent and Collaborative Research
    • Grants & Donations
• Investors & Media
  • Press Releases
  • Events & Presentations
  • Corporate Governance
    • Committee Composition
    • Contact the Board
  • Financial Information
    • SEC Filings
    • Annual Reports
    • Quarterly Results
    • Key Ratios
    • Financial Statements
  • Analyst Coverage
  • Stock Information
    • Historic Stock Lookup
  • Investor FAQs
  • Contact Us
  • Email Alerts
• 澳门正规博彩十大排行平台 Stories
• Clinical Trials
  • Hemophilia A
    • ROCTAVIAN™ (valoctocogene roxaparvovec-rvox)
  • Achondroplasia
    • VOXZOGO^® (vosoritide)
  • PKU
    • BMN 307 AAV PKU Gene Therapy
    • BMN 165 – Pegvaliase
    • KUVAN^® (sapropterin dihydrochloride)
    • PALYNZIQ^® (pegvaliase-pqpz) Injection
  • MPS IVA
  • MPS VI
  • CLN2 Disease
  • Hereditary Angioedema (HAE)
• Contact Us
  • Locations
  • Global Medical Information
  • Report Adverse Event
  • Report a Product Complaint
• Careers
• Privacy Policy
• Terms of Use
• Supplier Information
• Slavery Act Statement
• UK Tax Strategy
• Glossary